Screen push after stem cell success for babies with life-threatening immune system condition

STEM cell transplants are able to cure almost all babies born with a condition causing their immune system to malfunction, prompting calls to screen for the otherwise fatal disease.

A US study found up to 94 per cent of babies to get a transplant of blood-forming stem cells overcome severe combined immunodeficiency (SCID) — an inherited condition that stops their bodies fighting routine infections.

Newborns with the condition almost always die within a year if untreated; however, a US study of 240 babies receiving stem cell transplants found most were cured if they underwent the procedure in their first three-and-a-half months.

While the finding reinforces the success of the transplants already undertaken at Melbourne’s Royal Children’s Hospital, it has also led to a push for more widespread screening for SCID allowing doctors to administer treatment before it is too late.

Publishing his breakthrough in the New England Journal of Medicine, Dr Richard O’Reilly, from New York’s Memorial Sloan Kettering Cancer Centre, said the highest success rates occurred with babies able to gain a transplant from a matched sibling. “This confirms transplants for SCID work well in very young children, but it also shows any child with this disease can be treated with a high likelihood of a cure with a transplant from a parent or unrelated donor, not just a matched brother or sister,” Dr O’Reilly said. “Irrespective of the transplant approach used, if the child is transplanted early — without infection — you will have an extraordinarily good result.”

The RCH undertakes a handful of the transplants each year, but as between one in 65,000 and one in 100,000 Australian babies are believed to be born with the condition, the RCH’s Dr Francoise Mechinaud said the latest study should push practitioners to be aware and diagnose the condition earlier.